Amylyx Pharmaceuticals Receives U.S. FDA Fast Track Designation for AMX0114 for ALS

AMX0114 is an Amylyx-developed antisense oligonucleotide designed to target calpain-2, a key contributor to the axonal degeneration pathway in ALS

- Phase 1 LUMINA trial of AMX0114 underway; early cohort data expected in 2025

Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) (“Amylyx” or the “Company”) today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to AMX0114, an investigational antisense oligonucleotide (ASO) targeting calpain-2 for the treatment of people living with amyotrophic lateral sclerosis (ALS).

“Obtaining FDA Fast Track designation for AMX0114 is an important step forward in our mission to develop potential treatments for people living with ALS, a relentlessly progressive and fatal disease with limited therapeutic options,” said Camille L. Bedrosian, MD, Chief Medical Officer at Amylyx. “This designation from the FDA recognizes both the seriousness of this devastating disorder and the encouraging preclinical evidence supporting AMX0114’s potential to target calpain-2, which has been found to be an important contributor to axonal degeneration, a critical driver in ALS progression. We are committed to advancing AMX0114 as quickly and efficiently as possible, and we continue to anticipate early cohort data from the Phase 1 LUMINA clinical trial later this year. We look forward to continued interaction with the FDA as we work to expeditiously advance the development of AMX0114, with the ultimate goal of addressing the urgent, unmet needs of the ALS community.”

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